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FDA outlines collaborative approach to embracing real world data

Mar 28, 2019

The US Food and Drug Administration (FDA) has outlined a four-point plan to further embed real world evidence (RWE) into the regulatory pathway.

The Framework for Real-World Evidence Program will “apply a consistent strategy for harnessing” real world data (RWD) streams across the drug review process, says outgoing FDA commissioner Scott Gottlieb, M.D.

“Digital technologies are one of the most promising tools we have for making healthcare more efficient and more patient-focused,” he observed during a speech at the Bipartisan Policy Center conference.

“New streams of RWD gathered from electronic health records, lab tests, wearable devices, insurance claims, and even social media can provide important evidence on product safety and effectiveness in settings or populations that may be very different than the information gleaned from registration trials used for approval.”

Improving clinical trials

Dr. Gottlieb outlines four projects planned for 2019, all of which have been designed to advance the FDA’s use of RWE to complement clinical trials.

“First, to support the seamless integration of digital technologies in clinical trials, this year we plan to convene a stakeholder meeting to help develop a framework on how digital systems can be used to enhance the efficient oversight of clinical trials,” he says, adding that such technologies present important opportunities to streamline trials, such as remote monitoring.

“By working collaboratively with the clinical trial community and patient groups, we can develop scientific and technical standards for incorporating new technologies into clinical trials to make them more agile and accessible to patients and regulators.”

Creating more accessible, or de-centralized, clinical trials is another core part of the framework, notes Dr. Gottlieb. He adds that this could lead to more diverse participation as well as generate information that’s “more representative of the real world.”

A formal working group is currently working on a guidance document, expected later this year, that will outline the FDA’s approach to achieving this.

Analyzing change

The agency is also exploring ways in which reviewers could have more insight into how labeling changes inform provider prescribing decisions and patient outcomes.

Work by the Information Exchange and Data Transformation, or INFORMED, program, is an example of this. RWD is being used to analyze the impact of changing the dosing information on the FDA labels of two approved products.

In 2019, INFORMED will also be working with medical product centers and academics to develop an FDA curriculum on machine learning and artificial intelligence.

Investing in skills

The agency will also pilot a two-year artificial intelligence and machine learning fellowship program for post-doctoral fellows from leading academic centers.

Dr. Gottlieb says: “The aim of this program is to improve the ability of FDA reviewers and managers to evaluate products that incorporate advanced algorithms and facilitate the FDA’s capacity to develop novel regulatory science tools harnessing these approaches.”

Overall, the framework places an emphasis on working collaboratively with patients, patient advocacy groups and agencies such as the Centers for Medicare and Medicaid Services and the National Institutes of Health.

“By engaging with multiple stakeholders… the FDA can help promote more transparent standards for curating data, interoperability, and RWE generation that can help ensure that every American patient – no matter where they live – benefits from the full potential of these technologies,” concludes Dr. Gottlieb.

Astellas Patient Advocacy is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.